Joint bleeds in mild hemophilia: Prevalence and clinical characteristics.

INTRODUCTION: Joint bleeds are a common and frequent complication associated with hemophilia, increasing the risk of hemophilic arthropathy. It is important to define and characterize the presence of joint complications in mild hemophilia to develop strategies to mitigate disease burden. AIMS: To characterize the prevalence, clinical characteristics of joint bleeds, and risk factors that may lead to hemarthrosis in people with mild hemophilia. METHODS: Following Institutional Review Board approval, a retrospective chart review was conducted for patients with mild hemophilia seen at the Yale Hemophilia Treatment Center or Classical Hematology Program. RESULTS: The medical records of 70 patients were reviewed. Eighty one percent were male and 19 percent were female. Twenty individuals with mild hemophilia had a history of joint bleeding, 13 were traumatic bleeds, 7 were spontaneous. The age of first joint bleed ranged from 4 to 58 years old, with an average age of 20.8-years old. Ten patients developed joint bleeds between the ages of 10 and 20 years old. The most common locations of joint bleeding were the knee (n = 11) and ankle (n = 7). Eight of 70 patients had hepatitis C (HCV), 6 experienced joint bleeding. CONCLUSIONS: In this study, almost one third of patients with mild hemophilia experienced joint bleeding, often without history of trauma. Joint range of motion was abnormal in more than a third of the patients with mild hemophilia regardless. These data highlight the need for ongoing evaluation and characterization of joint health in individuals with mild hemophilia. HIGHLIGHTS: Twenty-nine percent of individuals with mild hemophilia had history of joint bleed. PwH and mild diseases with previous or current hepatitis C had higher likelihood of joint bleeding. Approximately 15% of PwH and mild diseases had abnormal joint examinations without a confirmed history of joint bleeding.

Current practices in pediatric hospital-acquired thromboembolism: Survey of the Children's Hospital Acquired Thrombosis (CHAT) Consortium.

Background: A rise in hospital-acquired venous thromboembolism (HA-VTE) in children has led to increased awareness regarding VTE prophylaxis and risk assessment. Despite no consensus exists regarding these practices in pediatrics. Objective: To describe common practices in VTE prophylaxis, VTE risk assessment models, and anticoagulation dosing strategies in pediatric hospitals that are members of the Children's Hospital Acquired Thrombosis (CHAT) Consortium. Methods: An electronic survey of 44 questions evaluating practices surrounding pediatric HA-VTE risk assessment and prevention was distributed between August 9, 2021, and August 30, 2021, to the primary investigators from the 32 institutions within the CHAT Consortium. Results: The survey response rate was 100% (n = 32). In total, 85% (n = 27) of the institutions assess HA-VTE, but only 63% (n = 20) have formal hospital guidelines. Within the institutions with formal guidelines, 100% (n = 20) include acute systemic inflammation or infection and presence of a central venous catheter (CVC) as risk factors for VTE. Pharmacologic prophylaxis is prescribed at 87% (28) of institutions, with enoxaparin being the most frequent (96%, n = 27). Variability in responses persisted regarding risk factors, risk assessment, thromboprophylaxis, dosing of prophylactic anticoagulation or anticoagulant drug monitoring. A majority of providers were comfortable providing thromboprophylaxis across all age groups. In addition, the global coronavirus disease 2019 increased the providers' use of prophylactic anticoagulation 78% (n = 25). Conclusion: Practices among institutions are variable in regard to use of HA-VTE prophylaxis, risk assessment, or guideline implementation, highlighting the need for further research and a validated risk assessment model through groups like the CHAT Consortium.

Practice patterns and real-life outcomes for patients with acute promyelocytic leukemia in the United States.

Acute promyelocytic leukemia (APL) is associated with a favorable long-term prognosis if appropriate treatment is initiated promptly. Outcomes in clinical trials and population-based registries vary; potential explanations include a delay in treatment and lower adherence to guideline-recommended therapy in real-world practice. We used the Vizient Clinical Data Base to describe demographic characteristics, baseline clinical characteristics, and treatment patterns in patients newly diagnosed with APL during the study period of April 2017 to March 2020. Baseline white blood cell count was used to assign risk status and assess treatment concordance with National Comprehensive Cancer Network guidelines. Logistic regression models examined adjusted associations between patient, hospital, disease characteristics, and adverse outcomes (in-hospital death or discharge to hospice). Among 1464 patients with APL, 205 (14.0%) experienced an adverse outcome. A substantial subset (20.6%) of patients did not receive guideline-concordant regimens. Odds of adverse outcomes increased with failure to receive guideline-concordant treatment (odds ratio [OR], 2.31; 95% confidence interval [CI], 1.43-3.75; P = .001), high-risk disease (OR, 2.48; 95% CI, 1.53-4.00; P < .001), and increasing age (≥60 years: OR, 11.13; 95% CI, 4.55-27.22; P < .001). Higher hospital acute myeloid leukemia (AML) patient volume was associated with lower odds of adverse outcome (OR, 0.44; 95% CI, 0.20-0.99 [for ≤50 vs >200 AML patients per year]; P = .046). In conclusion, in this large database analysis, 14.0% of patients newly diagnosed with APL died or were discharged to hospice. A substantial proportion of patients did not receive guideline-concordant therapy, potentially contributing to adverse outcomes.

Clinical decision rule for obtaining peripheral blood cultures in febrile oncology patients.

BACKGROUND: The utility of peripheral blood cultures in pediatric oncology patients presenting with fever is controversial. A recent systematic review showed that about one in 40 bloodstream infections (BSIs) would be missed if only central venous line (CVL) cultures are obtained. OBJECTIVE: To derive a clinical decision rule for obtaining peripheral blood cultures in pediatric oncology patients presenting to a pediatric emergency department (PED) with fever and a CVL. DESIGN/METHOD: A retrospective chart review was performed on pediatric oncology patients referred to the PED for fever while on therapy. Logistic regression with a random intercept was used to determine independent predictors of BSI and generate a prediction model for obtaining peripheral blood cultures. The decision rule was generated from the best performance as measured by a receiver operator curve. Bootstrapping analysis was performed for internal validation. RESULTS: Predictors that were significant and independently associated with positive peripheral blood cultures included vasopressor support (odds ratio [OR] 16.5, 95% confidence interval [CI]: 2.80-97.71), acute myeloid leukemia (AML) diagnosis (OR 6.9, 95% CI: 1.81-25.98), hypotension (OR 4.0, 95% CI: 1.05-15.17), mucositis (OR 8.2, 95% CI: 2.48-27.01), and maximum temperature in PED ≥39°C (OR 6.6, 95% CI: 2.36-18.20). The area under the curve (AUC) for this model was 0.90 (95% CI: 0.82-0.97) in the derivation cohort and 0.90 (95% CI: 0.81-0.98) after the internal validation. CONCLUSIONS: We derived a clinical prediction model for deciding when to obtain peripheral blood cultures in febrile oncology patients with CVLs on active therapy. Future studies should focus on prospective and external validation of this diagnostic prediction tool.

Patterns of medication use at end of life by pediatric inpatients with cancer.

OBJECTIVE: To describe medication utilization patterns by pediatric inpatients with cancer during their last week of life. METHODS: This retrospective study used data from the Vizient Clinical Database/Resource Manager, a national compilation of clinical and resource use data from over 100 academic medical centers and affiliates. Patients (0-21 years) with malignancy who died during hospitalization (2010-2017) were included (N = 1659). Medications were categorized as opioid, benzodiazepine, gastrointestinal related, chemotherapy, anti-infectives, or vasopressors. Exposure to each group was ascertained for all patients at 1 week and 1 day prior to death. Factors associated with exposure were examined using generalized estimating equations, and summarized using adjusted odds ratios (aORs). RESULTS: Over the last week of life, there was increased use of opioids (76% to 82%, aOR = 1.55, P < .001) and benzodiazepines (53% to 66%, aOR = 1.36, P = .02), while gastrointestinal-related medication use decreased (92% to 89%, aOR = 0.69, P = .001). Patients had decreased exposure to chemotherapy (10% to 5%, aOR = 0.46, P < .001) and anti-infectives (82% to 73%, aOR = 0.41, P = .002). Vasopressor use increased as death approached (15% to 28%, aOR = 1.67, P = .04). Factors significantly associated with exposure varied with medication category, and included age, race, length of stay, malignancy type, death in the intensive care unit, history of hematopoietic stem cell transplant, and do-not-resuscitate status. CONCLUSION: During the week preceding death, administration of symptom management medications increased for children with cancer, but use was not universal. Potentially life-sustaining medications were often continued. Variability in utilization suggests differences in provider/family decision making that warrant further study to develop an evidence-based approach to end-of-life care.

Assessing Advanced Communication Skills via Objective Structured Clinical Examination: A Comparison of Faculty Versus Self, Peer, and Standardized Patient Assessors.

Construct: The construct addressed in this study is assessment of advanced communication skills among senior medical students. Background: The question of who should assess participants during objective structured clinical examinations (OSCEs) has been debated, and options discussed in the literature have included peer, self, standardized patient, and faculty assessment models. What is not known is whether same-level peer assisted learning can be utilized for formative assessment of advanced communication skills when no faculty, standardized patients, or other trained assessors are involved in providing feedback. If successful, such an educational model would optimize resource utilization and broaden the scope of topics that could be covered in formative OSCEs. Approach: The investigators developed a 4-station formative OSCE focused on advanced communication skills for senior medical students, and evaluated the concordance of assessment done by same-level peers, self, standardized patients, and faculty for 45 students. After each station, examinees completed a self-assessment checklist and received checklist-based assessment and verbal feedback from same-level peers only. Standardized patients completed checklist-based assessments outside the room, and faculty did so after the OSCE via video review; neither group provided direct feedback to examinees. The investigators assessed inter-rater agreement and mean difference scores on the checklists using faculty score as the gold standard. Findings: There was fair to good overall agreement among self, same-level peer, standardized patient, and faculty-assessment of advanced communication skills. Relative to faculty, peer and standardized patient assessors overestimated advanced communication skills, while self-assessments underestimated skills. Conclusions: Self and same-level peer-assessment may be a viable alternative to faculty assessment for a formative OSCE on advanced communication skills for senior medical students.

Etoposide phosphate for pediatric orthopedic malignancies after intravenous etoposide hypersensitivity.

BACKGROUND: Hypersensitivity reactions to etoposide have been reported and patients have been safely transitioned to etoposide phosphate for continued therapy. However, the safety and efficacy of substituting etoposide phosphate for etoposide has not been well established in pediatric orthopedic malignancies. The aim of this study is to determine whether etoposide phosphate can be substituted for etoposide in pediatric orthopedic malignancies. METHODS: A chart review of pediatric patients who developed hypersensitivity reactions to etoposide while being treated for orthopedic malignancies was performed at a large academic medical center. Three patients were identified, two with Ewing sarcoma and one with an osteosarcoma. All three patients experienced hypersensitivity reactions to their first doses of etoposide and were switched to etoposide phosphate for further therapy. RESULTS: After premedication, all three patients tolerated full doses of etoposide phosphate without a graded dose challenge or desensitization. Two of the patients were premedicated with diphenhydramine alone, while the third received diphenhydramine and dexamethasone. CONCLUSIONS: Etoposide phosphate is a potentially safe alternative for pediatric patients with orthopedic malignancies who experience etoposide hypersensitivity. However, caution is needed as there are cases of etoposide phosphate hypersensitivity.

An Initiative to Decrease Time to Antibiotics for Patients With Fever and Neutropenia.

The objective was to decrease the time to antibiotic administration for patients arriving in the pediatric emergency department with fever and neutropenia. A multidisciplinary team was assembled and engaged in process analysis through interviews and data review. These findings were used to develop key drivers, and Pareto charts were utilized to prioritize interventions. Interventions were tested and implemented using rapid Plan-Do-Study-Act cycles. Progress was monitored using process control charts. Interventions included leveraging a secure text-based messaging platform, creating a new antibiotic pathway, and educating staff and family. Between September 2016 and September 2017, the average time to antibiotics was decreased from 116 to 55 minutes in this population. This also was associated with a decrease in variation (individual moving range mean decreased from 43 minutes to 18 minutes). Careful process analysis, coupled with the work of a multidisciplinary team, produced significant improvements in efficiency of care for these vulnerable patients.